The cure for some skin cancers and genetic diseases such as sickle-cell anemia may be within reach due to the monumental discovery and development of the genetic manipulation technology CRISPR-CAS9.

This technology holds the potential to knock such diseases out of the human genome forever, modifying genes more cheaply and accurately than ever before. The first human trials are set for this year.

“It’s revolutionary to me,” senior biology major Luis Popoca said, “the possibilities are just endless.”
But scientists quickly realized the dangers of this power over nature.

During a Science at 30 City talk on Feb. 19, PSUC biology professor Dr. Joel Parker urged the local community to think about how far this research should go. It was the 30th installment of Science at 30 City, a series of free science-related presentations usually held every first and third Monday at 30 City Hall Place.

“It’s going to be a slippery slope,” Parker said, “If you can cure genetic diseases, you can also make someone better. All the science fiction stuff is on the cusp of coming true.”
In 1975, as genetic modification gained traction, scientists flooded the sandy Asilomar Conference Grounds in Pacific Grove, California, to discuss the potential and dangers of biotechnology. They agreed that some experiments, such as cloning pathogens, shouldn’t be conducted.

“(Biologists) didn’t want to become the destroyers of the world like their physicist siblings,” Parker said.

More than a decade later, scientists discovered the basis of what was to be the CRISPR technology hidden in the endless battle between bacteria and viruses.

Bacteria evolved to encode short strands of viral DNA into their own, translate that into RNA, and use it to guide the CAS-9 protein in finding repeats of that DNA to eliminate. Eventually, scientists realized they could use this to target any genes they want, including those in humans, by specifying the guide RNA, Parker said. Genes can be turned off by CAS-9, and new genes can be added where others were removed. they named it clustered regularly interspaced palindromic repeats, or CRISPR. The technology continued to develop, and the first experiments began about 2012.

The development sparked more discussion on drawing guidelines for the future of CRISPR, resulting in the December, 2017, publishing of “Human Genome Editing: Science, Ethics, and Governance” by the National Academies of Sciences, Engineering and Medicine. The book, available as a free download, outlines the potentials and dangers of this relatively new technology. This time, scientists agreed not to use it for human enhancement.

Since 1975, “this is the closest thing to Asilomar,” Parker said.

Both Parker and Popoca noted the new possibilities of creating superhuman soldiers and athletes with boosted stamina. But CRISPR could also allow people to decide the genetic traits of their children.

“Maybe I want my kid to be taller, smarter, healthier,” Parker said. “Those questions are going to come up.”
Popoca said he’s concerned about the creation of an unbeatable virus.

The research has drawn criticism, particularly from the right-to-life and anti-abortion communities who believe life begins at conception. Many experiments have been conducted on zygotes — fertilized ova — left over from in vitro fertilization. Altering genes in human zygotes makes these alterations heritable, so “(We’re) talking about altering the human genome forever,” Parker said. Diseases such as Lou Gehrig’s could become problems of the past.

“(Right to life) is not going to stop China; it’s not going to stop Europe. They’re going to tear ahead with it.”

This year, French scientists will extract stem cells from someone with sickle-cell anemia, use CRISPR to activate fetal hemoglobin, which carries oxygen better than normal adult hemoglobin, and put those cells back.

“If they can get enough of those expressing, it should cure the disease,” Parker said.

Scientists at MIT this year will also use stem cells in trying to cure about 3 forms of skin cancer, altering stems cells to produce white blood cells that could attack the cancer.

Editing genes does carry the risk of creating mutations instead of curing them, Parker noted. But scientists are now focusing on engineering the guide RNA used by the CAS-9 protein to be more accurate by lengthening the target code, leaving less room for error.

Barrie Guibord, a history and social science faculty member at Clinton Community College said he’s “struck with the parallel discussion on artificial intelligence and how far you go with that.” He noted how Amish communities forbid things they perceive as harmful. “(With CRISPR,) we’re approaching that in a much larger way as a society,” he said.

This technology could reopen discussions about eugenics, and we need to start thinking about what is normal and how we define a genetic disease, Parker said.
“You can’t un-bite the apple; we’re heading into this full bore now. I don’t see any other way than to go down that path and see where it goes.”